Genetic Mechanisms in Neuroendocrine cell Hyperplasia of Infancy (NEHI)

MDBRNEHI

Feb 1

Written By Million Dollar Bike Ride

Awardee: Lisa Young

Institution: The Children's Hospital of Philadelphia

Award Amount: $60,989

MDBRNEHI2019

Million Dollar Bike Ride

Pre-clinical animal model and cell evaluation of candidate therapies for human NUBPL disease.

Tissue-specific and temporal reinstatement of Tcf4 to treat Pitt-Hopkins Syndrome